C. Developing and sharing validated targets

The industry must develop and share information about disease mechanisms and validated cellular targets to reduce redundant effort and to increase the chances of meaningful innovation.

Reisman’s comments initiated a conversation about the need for comprehensive data about medical outcomes that could inform effective decision making by consumers. Other panelists took the idea further, arguing that high-quality outcome information was a necessity not just for healthcare consumers, but for the creators of new drugs and devices as well. The path to an innovative solution requires an understanding of disease mechanisms and validated targets.

Multiple panelists and participants expressed concern about the time and expense necessary to move promising therapies through the clinical trials process, noting that current U.S. Food and Drug Administration (FDA) regulations impose obstacles that discourage investors and innovators. Eric S. Lander, President and Founding Director, The Eli and Edythe L. Broad Institute of Harvard and MIT, asked conference participants to reconsider the entire context of life sciences development. In his presentation, Lander said, “Really efficacious drugs don’t require huge clinical trials. Why aren’t we able to make drugs that are really efficacious rather than trying to demonstrate just a couple of percent benefit?”

“Really efficacious drugs don’t require huge clinical trials. Why aren’t we able to make drugs that are really efficacious rather than trying to demonstrate just a couple of percent benefit?”

President and Founding Director, The Eli and Edythe L. Broad Institute of Harvard and MIT

The group noted that basic knowledge, especially of the genome, has exploded over the previous ten years and that gene sequencing has become much cheaper as well. However, further discussion revealed that there are not good systems for developing validated targets and that targets are highly unlikely to be shared across organizations. Some suggested that in order to share targets consortia should be created, similar to what the semiconductor industry created in the 1980s and 1990s. If done correctly, this would create a critical mass of validated targets open to all, while preserving individual molecules as patentable intellectual property. The goal: an infrastructure that makes knowledge easily exchangeable.

Barbara J. McNeil, MD, Ridley Watts Professor at Harvard Medical School, underscored the important role that academic researchers can play in these collaborative efforts. “University investigators can play a key role in fostering innovation in a number of areas,” she said. “These range from developing new targets for drug discovery to working with others to develop disease-specific consortia, and finally to testing innovative products in pivotal clinical trials. Further along in the care pathway, they can use their clinical expertise to design and evaluate new approaches to the financing and delivering of healthcare, particularly coordinated care.”